Complex lethal osteochondrodysplasia

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ORPHA:457378OMIM:616897Q78.8
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Overview

Complex lethal osteochondrodysplasia is an extremely rare and severe bone and cartilage disorder that affects how the skeleton develops before birth. The term 'osteochondrodysplasia' refers to a group of conditions where bones and cartilage do not grow or form properly. When described as 'complex lethal,' it means the condition involves multiple severe skeletal abnormalities that are life-threatening, typically resulting in death before or shortly after birth. Babies with this condition may have very short limbs, an abnormally shaped chest that is too small to allow the lungs to work properly, underdeveloped bones, and other skeletal malformations. The small chest is often the most critical problem because it restricts breathing. Other features can include abnormal curvature of the spine, fractures present at birth, and poor mineralization of bones. Because this condition is so rare and severe, there is currently no cure or effective treatment that can change the outcome. Medical care focuses on comfort and supportive measures. Genetic counseling is important for affected families to understand the cause and the chance of recurrence in future pregnancies. Diagnosis is often made through prenatal ultrasound showing severe skeletal abnormalities, and may be confirmed with genetic testing when available.

Also known as:

Key symptoms:

Very short arms and legsAbnormally small chestSevere breathing difficultiesPoorly formed or soft bonesFractures present at birthAbnormal spine curvatureShort stature or dwarfismUnderdeveloped ribsAbnormal skull shapeReduced movement before birthLimb bowing

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

Treatments

No FDA-approved treatments are currently listed for Complex lethal osteochondrodysplasia.

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No actively recruiting trials found for Complex lethal osteochondrodysplasia at this time.

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Search ClinicalTrials.gov ↗Join the Complex lethal osteochondrodysplasia community →

No specialists are currently listed for Complex lethal osteochondrodysplasia.

View NORD Rare Disease Centers ↗Undiagnosed Disease Network ↗

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Complex lethal osteochondrodysplasia.

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Community

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Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

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Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Questions for your doctor

Bring these to your next appointment

  • Q1.What specific type of osteochondrodysplasia does my baby have, and is the exact genetic cause known?,What is the expected outcome, and what comfort care options are available?,Should we pursue genetic testing to identify the specific gene involved?,What is the chance this could happen again in a future pregnancy?,Are there prenatal testing options available for future pregnancies?,Can you connect us with palliative care and grief support services?,Are there any research studies or registries we could participate in?

Common questions about Complex lethal osteochondrodysplasia

What is Complex lethal osteochondrodysplasia?

Complex lethal osteochondrodysplasia is an extremely rare and severe bone and cartilage disorder that affects how the skeleton develops before birth. The term 'osteochondrodysplasia' refers to a group of conditions where bones and cartilage do not grow or form properly. When described as 'complex lethal,' it means the condition involves multiple severe skeletal abnormalities that are life-threatening, typically resulting in death before or shortly after birth. Babies with this condition may have very short limbs, an abnormally shaped chest that is too small to allow the lungs to work properly

At what age does Complex lethal osteochondrodysplasia typically begin?

Typical onset of Complex lethal osteochondrodysplasia is neonatal. Age of onset can vary across affected individuals.